Scientific leader who can independently design, execute, and guide effective experiments with clear goals in a very face paced environment.Work with the Cell-based assay team within CMC on the optimization, qualification/validation, and execution of phase-appropriate potency assays/lot release assays and broad spectrum of cell-based characterization assays (including immunoassays, nucleic acid-based assays, enzymatic assays, image-based based, among others).The position will have a strategic role in setting corporate direction and strategy for our bioassay and potency assay approach as well as a key operational role in day-to-day science and testing. He/She will have a leadership position within our growing QC team, and manage external collaborations (CRO, CDMO). The successful candidate will be experienced with development and validation of in-vitro potency assays for complex biologics and will drive the phase-appropriate method development, optimization, qualification and tech transfer. The position of Sr Manager/Sr Scientist, Potency Assays, will lead the QC arm of our cell-based assay group, overseeing the optimization and qualification of cell-based assays and bioassays for the lot release, characterization, and stability and formulation assessment of AAV-based gene therapy drug substance and drug product. Opportunities to expand to other indications and modalities within genetic medicine.Initial product safety and efficacy data substantiates the value of our platforms.Robust technology and IP foundation, including our TVE and manufacturing platforms.Five candidate products in the clinic and two declared pre-clinical programs.Demonstrated ability to move rapidly from idea to IND.Fully integrated clinical-phase company with internal manufacturing.This internal capability and close collaboration between our R&D and Manufacturing teams has greatly accelerated the pace of discovery at 4DMT.
In addition to TVE, our technology includes a robust AAV manufacturing platform and onsite manufacturing facility that allows us to rapidly produce and test research grade material and scale up to GMP clinical material.
We believe we are positioned to create, develop, manufacture and, if approved, effectively commercialize targeted genetic medicines that could transform the lives of patients suffering from debilitating diseases.
#4d molecular therapeutics salary trial
We have built a robust and efficient product engine with 6 open Investigational New Drug Applications (INDs) in the U.S., 1 IND in Taiwan, and 1 Clinical Trial Approval (CTA) in Australia. We believe this validates the power of our directed evolution platform for discovering superior vectors compared to wildtype viral vectors. To-date, we have demonstrated clinical proof-of-concept for three evolved vectors in three therapeutic areas and routes of administration with five products and patient populations. In addition, we have two product candidates in preclinical studies: 4D-175 for geographic atrophy (GA) and 4D-725 for alpha-1 antitrypsin deficiency. We have built a deep portfolio of AVV-based gene therapy product candidates, with five product candidates in clinical trials: 4D-150 for the treatment of wet age-related macular degeneration (wet AMD) and diabetic macular edema (DME), 4D-710 for the treatment of cystic fibrosis lung disease, 4D-310 for the treatment of Fabry disease cardiomyopathy, 4D-125 for the treatment of X-linked retinitis pigmentosa (XLRP), and 4D-110 for the treatment of choroideremia. These profiles will allow us to treat a broad range of large market diseases, unlike most current genetic medicines that generally focus on rare or small market diseases. We believe key features of our targeted and evolved vectors will help us create targeted product candidates with improved therapeutic profiles.
#4d molecular therapeutics salary full
We seek to unlock the full potential of gene therapy using our platform, Therapeutic Vector Evolution (TVE), which combines the power of directed evolution with our approximately one billion synthetic AAV capsid-derived sequences to invent evolved vectors for use in our products. We are committed to breaking boundaries and daring to cure as we develop new and foundational products and product components through our growing technology platforms.ĤDMT is a clinical-stage biotherapeutics company harnessing the power of directed evolution for targeted genetic medicines. (“4DMT”), we boldly innovate to unlock the full potential of genetic medicines for countless patients.
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